Section 5 of 6

Economic information and modeling report

11 evidence topics · 11 sources

Modeling overview

Summary

No published cost-effectiveness analysis, budget impact model, or health technology assessment of levacetylleucine in ataxia-telangiectasia (A-T) has been identified, and no source states a wholesale acquisition cost announced with the A-T approval of 18 September 2026.

IntraBio reported to California a wholesale acquisition cost of $13,450 per carton of 28 one-gram packets at introduction on 24 September 2024, and estimated 200 people in the United States with Niemann-Pick disease type C (NPC), the only approved indication at that time. An Oklahoma Medicaid review dated February 2025 listed $480.36 per packet and $691,718.40 per year at the maximum labeled dose of 4 g per day. The New York Medicaid list of reimbursable drugs effective 29 September 2026 carries a maximum reimbursable amount of $555.7142800 per packet. A September 2026 news report states a cost of $15,000 every one to two weeks.

National Medicaid data for the fourth quarter of 2025 record 5,852 units and $3,020,119.38 reimbursed under fee-for-service and 8,400 units and $4,381,592.98 under managed care. For the first quarter of 2026 the corresponding figures are 7,728 units and $4,017,756.66, and 6,076 units and $3,160,527.34. All of this utilization precedes the A-T approval.

IntraBio is privately held and has not disclosed sales. A January 2026 news report cites an analyst forecast of $500 million in peak U.S. sales in NPC; no forecast for the A-T indication was identified. An Oregon Medicaid evaluation dated December 2025, covering levacetylleucine and arimoclomol in NPC, graded the levacetylleucine evidence as low certainty, recommended non-preferred status with prior authorization, and listed levacetylleucine among the medications proposed for a carve-out of products with an estimated acquisition cost above $500,000 per member over 12 months.

For the eligible population, IntraBio states that A-T affects an estimated 1 in 40,000 people. Quince Therapeutics, the developer of another investigational A-T treatment, cites approximately 4,600 diagnosed people with A-T in the United States from IQVIA claims data.

Manufacturer estimate of the U.S. population at the 2024 launch: California filing

Field	Quoted record
Estimated number of patients	“200”
General comments	“This is a new and novel therapy for a disease called NPC Neimann Pick C. There were no previously approved indicated therapies for NPC.”

Medicaid utilization and reimbursement, national totals by quarter: 2025

Quarter and delivery system	Quoted units reimbursed	Quoted number of prescriptions	Quoted total amount reimbursed (USD)	Quoted Medicaid amount reimbursed (USD)
First quarter 2025, managed care	“2604.000”	“33”	“1264504.78”	“1264504.78”
Second quarter 2025, fee-for-service	“3080.000”	“36”	“1454153.49”	“1395177.93”
Second quarter 2025, managed care	“4872.000”	“61”	“2416559.79”	“2316942.59”
Third quarter 2025, fee-for-service	“6552.000”	“79”	“3288367.91”	“3196467.73”
Third quarter 2025, managed care	“6860.000”	“84”	“3530837.46”	“3413044.04”
Fourth quarter 2025, fee-for-service	“5852.000”	“69”	“3020119.38”	“2922717.47”
Fourth quarter 2025, managed care	“8400.000”	“101”	“4381592.98”	“4193836.02”

Medicaid utilization and reimbursement, national totals: first quarter 2026

Quarter and delivery system	Quoted units reimbursed	Quoted number of prescriptions	Quoted total amount reimbursed (USD)	Quoted Medicaid amount reimbursed (USD)
First quarter 2026, fee-for-service	“7728.000”	“93”	“4017756.66”	“4017744.66”
First quarter 2026, managed care	“6076.000”	“70”	“3160527.34”	“2874948.28”

Company sales disclosure and analyst forecast

“IntraBio, a private company, has not disclosed sales figures for Aqneursa, but Zevra has secured 137 prescription enrollment forms since its NPC launch as of the third quarter.” (opens the source at this quote in a new tab)
“Analysts at Delveinsight expect Aqneursa to generate $500 million in peak U.S. sales in NPC.” (opens the source at this quote in a new tab)

Eligible population: prevalence stated by the company at approval

“A-T is a rare, progressive, and prematurely fatal autosomal-recessive neurodegenerative disorder that affects an estimated 1 in 40,000 people.” (opens the source at this quote in a new tab)

Eligible population: diagnosed U.S. patients cited by another developer

“Based on IQVIA Medical Claims (Dx), PharmetricsPlus (P+), and IQVIA Analytics information, there are approximately 4,600 diagnosed patients with A-T in the U.S.” (opens the source at this quote in a new tab)

Oregon Medicaid new drug evaluation in Niemann-Pick disease type C: scope

“New Drug Evaluations: Miplyffa™ (arimoclomol) capsules, Aqneursa™ (levacetylleucine) oral suspension” (opens the source at this quote in a new tab)
“Date of Review: December 2025” (opens the source at this quote in a new tab)
“Dossier Received: MIPLYFFA (yes); AQNEURSA (no)” (opens the source at this quote in a new tab)

Oregon Medicaid new drug evaluation in Niemann-Pick disease type C: evidence conclusions

“There is low-certainty evidence from one randomized, double-blind, phase III, crossover trial that levacetylleucine improves ataxia symptoms over 12 weeks in patients 4 years of age and older with NPC (n=60) weighing at least 15 kg.” (opens the source at this quote in a new tab)
“There is no comparative data between treatments of arimoclomol with miglustat versus levacetylleucine.” (opens the source at this quote in a new tab)

Oregon Medicaid new drug evaluation in Niemann-Pick disease type C: recommendations

“Make arimoclomol and levacetylleucine non-preferred on the Oregon Health Plan (OHP) Preferred Drug list (PDL).” (opens the source at this quote in a new tab)
“Implement prior authorization (PA) for high cost treatments for Niemann-Pick disease type C.” (opens the source at this quote in a new tab)

Oregon Medicaid new drug evaluation in Niemann-Pick disease type C: high-cost carve-out and affected members

“Beginning in January 2026, the Oregon Health Authority is proposing that high cost medications for rare conditions be carved out of Coordinated Care Organization (CCO) payments and billed directly to fee-for-service (FFS).” (opens the source at this quote in a new tab)
“Estimated acquisition cost of more than $500,000 per member over a 12-month period” (opens the source at this quote in a new tab)
“Both arimoclomol and levacetylleucine are currently included in the list of medications proposed to be carved-out of CCO budgets.” (opens the source at this quote in a new tab)
“Over a 1 year period from 4/1/24 to 3/31/25, there were 8 Oregon Health Plan members with a diagnosis indicating NPC in their medical claims.” (opens the source at this quote in a new tab)

Budget impact model

Approach and framework

No evidence found.

Perspective and time frame

No evidence found.

Epidemiology and eligible population inputs

No evidence found.

Cost assumptions

No evidence found.

Model outcomes

No evidence found.

Results

Base case

No evidence found.

Scenario analyses

No evidence found.

Budget impact model discussion

No evidence found.